Targeting Metabolic Dysfunction Underlying Diseases of Aging
| Target Indication | Preclinical | IND Enabling Activities | Clinical Phase 1 | |
|---|---|---|---|---|
| Discovery | Optimization | |||
| MOTS-c Analog CB4211 | 2 Targets | |||
| NASH |
Discovery Phase complete
|
Optimization Phase complete
|
IND Enabling Activities Phase in progress
|
Clinical Phase 1 Phase not started
|
| Obesity |
Discovery Phase complete
|
Optimization Phase complete
|
IND Enabling Activities Phase in progress
|
Clinical Phase 1 Phase not started
|
| New MDP Analogs | 5 Targets | |||
| Obesity/NASH |
Discovery Phase complete
|
Optimization Phase in progress
|
IND Enabling Activities Phase not started
|
Clinical Phase 1 Phase not started
|
| Type 2 diabetes |
Discovery Phase complete
|
Optimization Phase in progress
|
IND Enabling Activities Phase not started
|
Clinical Phase 1 Phase not started
|
| Cancer |
Discovery Phase in progress
|
Optimization Phase not started
|
IND Enabling Activities Phase not started
|
Clinical Phase 1 Phase not started
|
| Neurodegenerative Diseases |
Discovery Phase in progress
|
Optimization Phase not started
|
IND Enabling Activities Phase not started
|
Clinical Phase 1 Phase not started
|
| Cardiovascular Disease |
Discovery Phase in progress
|
Optimization Phase not started
|
IND Enabling Activities Phase not started
|
Clinical Phase 1 Phase not started
|
Lead Program, CB4211
CB4211 is a novel, optimized analog of MOTS-c, a naturally occurring mitochondrial peptide discovered by our founder and his academic collaborator in 2012. Their research in cells and animal models indicated that MOTS-c plays a significant role in the regulation of metabolism. Certain of the original MOTS-c studies were published in an article entitled "The Mitochondrial-Derived Peptide, MOTS-c, Promotes Metabolic Homeostasis and Reduces Obesity and Insulin Resistance," which appeared in the March 3, 2015 edition of the journal Cell Metabolism.
In pre-clinical models, CB4211 has demonstrated significant therapeutic potential for the treatment of NASH, showing improvements in triglyceride levels, as well as favorable effects on liver enzyme markers associated with NAFLD and NASH, and obesity, demonstrating significantly greater weight loss together with more selective reduction of fat mass versus lean mass in head-to-head comparison to a market-leading obesity drug.
The therapeutic effects of CB4211 have been further evaluated in the well-established preclinical STAM™ mouse model of NASH. In this model, treatment with CB4211 resulted in a significant reduction of the non-alcoholic fatty liver disease activity score, or NAS, a composite measure of steatosis (fat accumulation), inflammation and hepatocyte ballooning (cellular injury).
New MDP Analogs
Our R&D pipeline also includes a number of newly discovered mitochondrial-derived peptides (MDPs) and their analogs. Pre-clinical activities with respect to these peptides are focused on identifying and optimizing those MDPs and their analogs that demonstrate the greatest commercial and therapeutic potential as mitochondria based therapeutics (MBTs). These MDPs and their analogs have demonstrated various degrees of biological activity in a wide range of cell based and/or animal models relevant to diseases, such as NASH, obesity, T2D, cancer, cardiovascular disease and Alzheimer's disease.